Publications & Presentations

Get key insights from published articles, posters, and other rare disease research from our team of clinicians and scientists.

International Society for the Study of Vascular Anomalies (ISSVA) World Congress 2026

QTORIN™ 3.9% Rapamycin Anhydrous Gel: Statistically Significant, Clinically Meaningful Improvement in Microcystic Lymphatic Malformations (Phase 3 SELVA Study) and Cutaneous Venous Malformations (Phase 2 TOIVA Study)
Presenter: James Treat, MD, Professor of Clinical Pediatrics and Dermatology at the Children’s Hospital of Philadelphia
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Society for Investigative Dermatology (SID) 2026 Annual Meeting

Significant reductions in lesion bleeding with QTORIN™ 3.9% rapamycin in cutaneous venous malformations
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
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12th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference

SELVA: A Phase 3 Study with a Fit-For-Purpose Primary Endpoint Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations
Presenter: Joyce Teng, M.D., Ph.D.; Professor of Dermatology and Pediatrics, Stanford University
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QTORINTM rapamycin 3.9% anhydrous gel: A Novel, Dermal-Targeted Topical mTOR Inhibitor in Phase 3 for Treatment of Microcystic Lymphatic Malformations
Presenter: Jeff Martini, Ph.D., Chief Scientific Officer, Palvella Therapeutics
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Rare Genodermatoses

Licholai G. Rare Dermatology: Orphan Disease Drug Development Opportunities and Challenges. Dermatology Summit (2019).
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Doux J. Barriers and Opportunities: A View Across the Developmental Divide. Journal of Investigative Dermatology (2015) 135, 2143–2144.
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Microcystic Lymphatic Malformations

Palvella Therapeutics, Inc. Microcystic and Macrocystic Lymphatic Malformations: Distinct Genetics and Clinical Strategies. Journal of Vascular Anomalies (2026).
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Teng JMC, et. al., Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphatic Research and Biology (2022).
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Carroll S, et. al., Annual prevalence estimation of lymphatic malformation with a cutaneous component: observational study of a national representative sample of physicians. Orphanet Journal of Rare Diseases (2022).
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Canaud G, et al., A review of mechanisms of disease across PIK3CA-related disorders with vascular manifestations. Orphanet Journal of Rare Diseases (2021).
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Adams DM, et al., Efficacy and Safety of Sirolimus in the Treatment of Complicated Vascular Anomalies. Pediatrics (2016).
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Hammill AM, et al., Sirolimus for the treatment of complicated vascular anomalies in children. Pediatric Blood and Cancer (2011).
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